PMID- 31859676 OWN - NLM STAT- MEDLINE DCOM- 20200915 LR - 20230502 IS - 2291-5222 (Electronic) IS - 2291-5222 (Linking) VI - 7 IP - 12 DP - 2019 Dec 20 TI - Objectively Monitoring Amyotrophic Lateral Sclerosis Patient Symptoms During Clinical Trials With Sensors: Observational Study. PG - e13433 LID - 10.2196/13433 [doi] LID - e13433 AB - BACKGROUND: Objective symptom monitoring of patients with Amyotrophic Lateral Sclerosis (ALS) has the potential to provide an important source of information to evaluate the impact of the disease on aspects of real-world functional capacity and activities of daily living in the home setting, providing useful objective outcome measures for clinical trials. OBJECTIVE: This study aimed to investigate the feasibility of a novel digital platform for remote data collection of multiple symptoms-physical activity, heart rate variability (HRV), and digital speech characteristics-in 25 patients with ALS in an observational clinical trial setting to explore the impact of the devices on patients' everyday life and to record tolerability related to the devices and study procedures over 48 weeks. METHODS: In this exploratory, noncontrolled, nondrug study, patients attended a clinical site visit every 3 months to perform activity reference tasks while wearing a sensor, to conduct digital speech tests and for conventional ALS monitoring. In addition, patients wore the sensor in their daily life for approximately 3 days every month for the duration of the study. RESULTS: The amount and quality of digital speech data captured at the clinical sites were as intended, and there were no significant issues. All the home monitoring sensor data available were propagated through the system and were received as expected. However, the amount and quality of physical activity home monitoring data were lower than anticipated. A total of 3 or more days (or partial days) of data were recorded for 65% of protocol time points, with no data collected for 24% of time points. At baseline, 24 of 25 patients provided data, reduced to 13 of 18 patients at Week 48. Lower-than-expected quality HRV data were obtained, likely because of poor contact between the sensor and the skin. In total, 6 of 25 patients had mild or moderate adverse events (AEs) in the skin and subcutaneous tissue disorders category because of skin irritation caused by the electrode patch. There were no reports of serious AEs or deaths. Most patients found the sensor comfortable, with no or minimal impact on daily activities. CONCLUSIONS: The platform can measure physical activity in patients with ALS in their home environment; patients used the equipment successfully, and it was generally well tolerated. The quantity of home monitoring physical activity data was lower than expected, although it was sufficient to allow investigation of novel physical activity end points. Good-quality in-clinic speech data were successfully captured for analysis. Future studies using objective patient monitoring approaches, combined with the most current technological advances, may be useful to elucidate novel digital biomarkers of disease progression. CI - (c)Luis Garcia-Gancedo, Madeline L Kelly, Arseniy Lavrov, Jim Parr, Rob Hart, Rachael Marsden, Martin R Turner, Kevin Talbot, Theresa Chiwera, Christopher E Shaw, Ammar Al-Chalabi. Originally published in JMIR mHealth and uHealth (http://mhealth.jmir.org), 20.12.2019. FAU - Garcia-Gancedo, Luis AU - Garcia-Gancedo L AUID- ORCID: 0000-0001-5631-1711 AD - Advanced Biostatistics & Data Analytics Centre of Excellence, R&D Projects Clinical Platforms & Sciences, GlaxoSmithKline, Stevenage, United Kingdom. FAU - Kelly, Madeline L AU - Kelly ML AUID- ORCID: 0000-0001-6855-4550 AD - Translational Medicine, Future Pipeline Discovery, GlaxoSmithKline, Stevenage, United Kingdom. FAU - Lavrov, Arseniy AU - Lavrov A AUID- ORCID: 0000-0002-2360-2253 AD - Clinical Development, AveXis, Bannockburn, IL, United States. FAU - Parr, Jim AU - Parr J AUID- ORCID: 0000-0001-5956-217X AD - McLaren Technology Centre, McLaren Applied Technologies, Woking, United Kingdom. FAU - Hart, Rob AU - Hart R AUID- ORCID: 0000-0002-8507-3326 AD - McLaren Technology Centre, McLaren Applied Technologies, Woking, United Kingdom. FAU - Marsden, Rachael AU - Marsden R AUID- ORCID: 0000-0003-0304-9427 AD - Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom. FAU - Turner, Martin R AU - Turner MR AUID- ORCID: 0000-0003-0267-3180 AD - Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom. FAU - Talbot, Kevin AU - Talbot K AUID- ORCID: 0000-0001-5490-1697 AD - Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom. FAU - Chiwera, Theresa AU - Chiwera T AUID- ORCID: 0000-0003-2884-4267 AD - Maurice Wohl Clinical Neuroscience Institute, Department of Basic and Clinical Neuroscience, King's College London, London, United Kingdom. FAU - Shaw, Christopher E AU - Shaw CE AUID- ORCID: 0000-0003-0251-0146 AD - Maurice Wohl Clinical Neuroscience Institute, Department of Basic and Clinical Neuroscience, King's College London, London, United Kingdom. FAU - Al-Chalabi, Ammar AU - Al-Chalabi A AUID- ORCID: 0000-0002-4924-7712 AD - Maurice Wohl Clinical Neuroscience Institute, Department of Basic and Clinical Neuroscience, King's College London, London, United Kingdom. LA - eng GR - ALCHALABI-TALBOT/APR14/926-794/MNDA_/Motor Neurone Disease Association/United Kingdom GR - G0500289/MRC_/Medical Research Council/United Kingdom GR - MC_PC_17115/MRC_/Medical Research Council/United Kingdom GR - TURNER/OCT18/989-797/MNDA_/Motor Neurone Disease Association/United Kingdom GR - DH_/Department of Health/United Kingdom PT - Clinical Trial PT - Journal Article PT - Observational Study PT - Research Support, Non-U.S. Gov't DEP - 20191220 PL - Canada TA - JMIR Mhealth Uhealth JT - JMIR mHealth and uHealth JID - 101624439 SB - IM MH - Activities of Daily Living MH - Adult MH - Amyotrophic Lateral Sclerosis/*diagnosis/ethnology MH - Data Collection/*methods MH - Disease Progression MH - Exercise/physiology MH - Feasibility Studies MH - Female MH - Heart Rate/physiology MH - Humans MH - Male MH - Middle Aged MH - Monitoring, Physiologic/*instrumentation MH - Phenotype MH - Speech/physiology MH - Technology MH - Wearable Electronic Devices/*adverse effects PMC - PMC6942190 OTO - NOTNLM OT - accelerometer OT - amyotrophic lateral sclerosis OT - clinical trial OT - digital biomarker OT - digital phenotyping OT - heart rate OT - objective symptom monitoring OT - physical activity OT - speech OT - wearable COIS- Conflicts of Interest: LG-G and MLK are employees of GSK and hold stocks/shares. At the time of the study, AL was an employee of GSK; currently, he holds stocks/shares. CES has previously consulted for GSK (>5 years ago), has received research grants from Vertex and Chronos Therapeutics in the past, and has an active grant with Eli Lily. AA-C reports consultancies for GSK, Cytokinetics, Biogen Idec, Treeway Inc, Chronos Therapeutics, OrionPharma, and Mitsubishi-Tanabe Pharma and was chief investigator for commercial clinical trials run by OrionPharma and Cytokinetics. JP, RH, RM, MRT, KT, and TC have no conflicts of interest to declare. EDAT- 2019/12/21 06:00 MHDA- 2020/09/17 06:00 PMCR- 2019/12/20 CRDT- 2019/12/21 06:00 PHST- 2019/01/18 00:00 [received] PHST- 2019/09/26 00:00 [accepted] PHST- 2019/07/11 00:00 [revised] PHST- 2019/12/21 06:00 [entrez] PHST- 2019/12/21 06:00 [pubmed] PHST- 2020/09/17 06:00 [medline] PHST- 2019/12/20 00:00 [pmc-release] AID - v7i12e13433 [pii] AID - 10.2196/13433 [doi] PST - epublish SO - JMIR Mhealth Uhealth. 2019 Dec 20;7(12):e13433. doi: 10.2196/13433.